Baxalta Announces Priority Review Status Granted By Health Canada For Irinotecan Liposome Injection (Nal-IRI) For New Drug Submission

BANNOCKBURN, Ill.--(BUSINESS WIRE)--Baxalta Incorporated (NYSE: BXLT), a global biopharmaceutical leader dedicated to delivering transformative therapies to patients with orphan diseases and underserved conditions, today announced that Health Canada has granted Priority Review status for its New Drug Submission (NDS) for irinotecan liposome injection, also known as "nal-IRI". Baxalta is seeking marketing approval of nal-IRI from Health Canada for the treatment of patients with metastatic adenocarcinoma of the pancreas previously treated with gemcitabine-based therapy.

Priority Review allows for expedited review of critical new drugs faster than the standard timeline. Review by Health Canada is expected to be conducted in the second half of 2016. Baxalta is responsible for the development and commercialization of nal-IRI outside of the U.S. and Taiwan under the exclusive licensing agreement with Merrimack Pharmaceuticals, Inc.

“We are encouraged by the decision Health Canada has made to review our NDS of nal-IRI on an expedited timeline,” said Salim Yazji, M.D., vice president and global therapeutics head, Oncology, Baxalta. “Pancreatic cancer is one of the few cancers for which survival has not improved substantially over the last few decades, largely due to late diagnosis, aggressiveness of the disease and limited treatment options. This is an important milestone for our joint efforts to bring nal-IRI to more patients worldwide and fight this devastating cancer of the pancreas to help fill a significant unmet medical need.”

The application is based upon the results of an international Phase 3 study (NAPOLI-1) conducted among patients with metastatic pancreatic cancer (mPaC) who previously received gemcitabine-based therapy. In combination with 5-fluorouracil (5-FU) and leucovorin (LV), nal-IRI achieved its primary and secondary endpoints by demonstrating a statistically significant improvement in overall survival, progression free survival and overall response rate compared to the control group of patients who received a combination of 5-FU and LV. The most common Grade 3 or higher adverse events in patients receiving nal-IRI and 5-FU/LV were neutropenia, fatigue and gastrointestinal effects. This was the first global Phase 3 study in a post-gemcitabine setting to demonstrate a survival benefit in the treatment of this aggressive disease.

Baxalta and Merrimack recently enrolled the first patient in an exploratory Phase 2 clinical study of nal-IRI in previously untreated, metastatic pancreatic adenocarcinoma to assess the safety and efficacy of the combination of nal-IRI, 5-FU and LV, with or without the addition of oxaliplatin, versus nab-paclitaxel and gemcitabine. Based on the current design of this study, data is expected in the first half of 2017. In addition to studying nal-IRI in patients with pancreatic cancer, there are ongoing Phase 1 studies of nal-IRI in patients with pediatric sarcoma, glioma, breast and gastric cancer.

A Marketing Authorization Application submitted by Baxalta is also under review in the European Union for the treatment of adult patients with metastatic adenocarcinoma of the pancreas who have previously been treated with gemcitabine-based therapy. The decision from Health Canada follows the recent U.S. Food and Drug Administration (FDA) approval of nal-IRI, Merrimack’s (Onivyde) New Drug Application for the same indication using the same clinical efficacy and safety data.

About Pancreatic Cancer

Pancreatic cancer is rare and deadly - accounting for less than three percent of all cancer cases worldwide, but the seventh leading cause of cancer death¹. An estimated 340,000 new cases are diagnosed every year around the world¹, about two-thirds of which are among people aged 65 or older². It is estimated that there were 4,800 new cases of pancreatic cancer this past year in Canada specifically³.

Because the signs and symptoms of pancreatic cancer are non-specific and may not appear until the disease has spread to other sites, approximately 80 percent of patients are not candidates for surgery; these patients instead receive chemotherapy as the mainstay of their therapy⁴. This contributes to the five-year survival rate for all patients being less than six percent⁵. There is no consensus on the standard of care for patients with mPaC previously treated with a gemcitabine-based therapy.

About irinotecan liposome injection (nal-IRI)

nal-IRI is an investigational nanoliposome consisting of the chemotherapeutic, irinotecan, encapsulated in a liposomal sphere. Merrimack received FDA approval for nal-IRI under the brand name Onivyde™ for the treatment of patients with metastatic adenocarcinoma of the pancreas who have been previously treated with gemcitabine-based therapy. nal-IRI was granted Fast Track designation and Priority Review by the FDA for this indication. The FDA also granted nal-IRI orphan drug designation for patients with mPaC. Baxalta is responsible for the development and commercialization of nal-IRI outside of the United States and Taiwan under an exclusive licensing agreement with Merrimack. PharmaEngine holds the rights to commercialize Onivyde in Taiwan, following TFDA approval in October 2015.

About Baxalta

Baxalta Incorporated (NYSE: BXLT) is a $6 billion global biopharmaceutical leader developing, manufacturing and commercializing therapies for orphan diseases and underserved conditions in hematology, oncology and immunology. Driven by passion to make a meaningful impact on patients’ lives, Baxalta’s broad and diverse pipeline includes biologics with novel mechanisms and advanced technology platforms such as gene therapy. The Baxalta Global Innovation and R&D Center is located in Cambridge, Massachusetts. Launched in 2015 following separation from Baxter International, Baxalta’s heritage in biopharmaceuticals spans decades. Baxalta’s therapies are available in more than 100 countries and it has advanced biological manufacturing operations across 12 facilities, including state-of-the-art recombinant production and plasma fractionation. Headquartered in Northern Illinois, Baxalta employs 16,000 employees worldwide.

Forward-Looking Statements

This release includes forward-looking statements concerning nal-IRI, including expectations with regard to clinical studies, regulatory filings and potential impact to patients. Such statements are made as of the date that they were first issued and are based on current expectations, beliefs and assumptions of management. Forward-looking statements are subject to a number of risks and uncertainties, many of which involve factors or circumstances that are beyond Baxalta's control and which could cause actual results to differ materially from those in the forward-looking statements, including the following: clinical trial results; satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; product quality, manufacturing or supply issues; patient safety issues; and other risks identified in Baxalta's filings with the Securities and Exchange Commission, all of which are available on Baxalta’s website. Baxalta expressly disclaims any intent or obligation to update these forward-looking statements except as required by law.

References

1. International Agency for Research on Cancer, World Health Organization (website). GLOBOCAN 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide in 2012. Accessed December 11, 2015. Available at: http://globocan.iarc.fr/Pages/fact_sheets_population.aspx
2. American Cancer Society (website). Pancreatic Cancer: Causes, Risk Factors, And Prevention Topics. Accessed December 11, 2015. Available at: http://www.cancer.org/cancer/pancreaticcancer/detailedguide/pancreatic-cancer-risk-factors
3. Public Health Agency of Canada, Canadian Cancer Society (website). Canadian Cancer Statistics 2015. Accessed December 11, 2015. Available at: https://www.cancer.ca/~/media/cancer.ca/CW/cancer%20information/cancer%20101/Canadian%20cancer%20statistics/Canadian-Cancer-Statistics-2015-EN.pdf
4. Pancreatic Cancer Action Network (website). Pancreatic Cancer Facts 2015. Accessed December 11, 2015. Accessed: https://www.pancan.org/wp-content/uploads/2015/06/2015-GAA-PC-Facts.pdf
5. American Cancer Society (website). Cancer Facts and Figures 2014. Accessed December 11, 2015. Available at: http://www.cancer.org/acs/groups/content/@research/documents/webcontent/acspc-042151.pdf